A step forward, but not the finish line: What NICE’s QALY update means for rare diseases

Healthcare in the UK is at an inflection point. To ensure the NHS remains fit for the future, the Government launched its 10-Year Health Plan for England last year, setting out strong ambitions around prevention, digitisation, and community care. However, despite the pivotal role medicines can play in achieving transformation – from improving population health to reducing long-term demand on services – investment in medicines has been largely stagnant in recent years. But policy reform must translate into tangible improvements for the millions of people affected by rare conditions in the UK.

Against this backdrop, the government’s decision at the end of last year to raise NICE’s standard cost-effectiveness threshold for appraising new medicines from £20,000-£30,000 to between £25,000-£35,000 per quality adjusted life year (QALY) gained is welcome.[1] The move, set to come into effect from this month onwards, marks the first change to the threshold since NICE’s inception and has been framed as supporting access to innovative medicines.

While positive, for rare disease it represents the first step of a much longer journey – one that will determine whether groundbreaking treatments reach patients, whether the UK remains globally competitive, and ultimately whether the NHS can continue delivering on its founding principle of equity.

Why the change matters

The increase in NICE’s standard cost-effectiveness threshold highlights a greater willingness to pay for innovation and reflects growing recognition that the previous framework had become misaligned with the value of modern medicines. The rise is predicted to enable an additional three to five medicines or indications to be recommended each year, which is good news for patients waiting for new treatment options.[1]

The decision also serves as an important market signal. Global companies factor reimbursement expectations into their launch planning: if the UK is seen as unlikely to approve or adequately reimburse a therapy, it risks being deprioritised in favour of countries with more predictable and flexible processes. A higher probability of a positive NICE appraisal could help shift that dynamic, boosting inward investment, clinical trial activity, and scientific engagement.

Spotlighting the remaining challenge for rare disease

Rare disease therapies often require disproportionately high investment relative to the size of the patient population. As a result, HTA bodies must be willing to pay a higher price to provide equitable care for people living with rare diseases. While the increase to NICE’s standard threshold moves us in the right direction, the fact remains that many rare disease treatments will continue to be deemed not cost-effective for NHS use.

One fundamental challenge is many therapies for rare diseases tend to sit awkwardly between NICE’s Standard Technology Appraisal (STA) route (where the upper QALY is set to be £35,000 soon) and the Highly Specialised Technology (HST) pathway reserved for ultra-rare, severe, and chronic diseases (where the threshold can be up to £300,000 per QALY gained). Falling between the gaps in assessment frameworks can mean that treatments never reach the people who need them.

Conventional HTA models often struggle with rare diseases for another reason, too. These frameworks favour robust certainty as a core component of the decision-making process. NICE, for example, aims to provide evidence-based recommendations on the value of medicines, often requiring high levels of certainty regarding clinical- and cost-effectiveness to ensure health system sustainability. Rare disease treatments, on the other hand, are often tested in smaller patient numbers in clinical trials and have less supporting data. This inherent uncertainty is the reality of rare disease research and should not be seen as a reflection of weaker science or lower patient need.

The culmination of these issues means medicines for rare diseases frequently fail to meet standard cost-effectiveness criteria. Despite collectively impacting an estimated 3.5 million people in the UK, only one in 20 of the approximately 7,000 known rare diseases has a treatment available on the NHS.[2]^,[3]

What needs to happen in rare disease

We can look to other countries for inspiration on how to further enhance access to rare disease treatments. In Sweden, the HTA review of medicines for rare diseases applies explicit ethical principles alongside cost-effectiveness, giving greater weight to severity, unmet need, and lack of alternatives.[4] This approach means a higher cost per QALY and greater uncertainty can be accepted for rare diseases, translating into fairer medicines access.

Similarly in Spain, the publication of the “HTA Royal Decree” process gives consideration to multiple criteria beyond cost-effectiveness, including disease severity, ethics, and social factors.[5] These approaches recognise that value is multidimensional, especially for small, underserved patient populations.

While the updated NICE threshold for cost-effectiveness is an important step forward, it does not fundamentally change how value is assessed. Instead, for the assessment of rare disease therapies, there should be a broader set of criteria that doesn’t solely focus on health economic modelling to make appraisal decisions. This model should acknowledge the realities of rare disease R&D and place clinical need, societal benefit, and equity alongside cost effectiveness.

Meaningful progress requires political will and the recognition that society must accept a higher level of investment where equity of access is at stake – an investment that pays back through long-term economic benefits, reduced disease burden and, ultimately, transformed lives.

The UK has huge potential

The UK is home to world-leading universities, globally recognised academic institutions, and highly engaged patient communities. These strengths create a significant opportunity for the country to lead in rare disease research.

At its core, the NHS is founded on a belief in equity and the view that no one should be disadvantaged because of who they are or the condition they have. Rarity should never be a reason for exclusion from care, and yet we have seen the rare disease community in the UK miss out on access to important medicines time and again. Recent decisions on medicines for acid sphingomyelinase deficiency (Niemann-Pick disease) and achondroplasia, illustrate how patients can face prolonged delays or restricted access despite strong clinical value and high unmet need.[6]^,[7]

While NICE’s updated QALY range is welcome and has the potential to improve the lives of people with a rare disease, the framework for assessing new medicines must be accompanied by a broader set of criteria that reflect the realities of rare disease research.

With the right execution, commercial flexibility and policy alignment, the UK has a chance to not only improve patient access, but to re-emerge as a global leader in rare disease innovation. The opportunity is there, what’s needed now is the courage and commitment to seize it.

References

[1] NICE. Changes to NICE’s cost-effectiveness thresholds confirmed. Available at: https://www.nice.org.uk/news/articles/changes-to-nice-s-cost-effectiveness-thresholds-confirmed. Accessed: February 2026.

[2] NIHR. UK rare disease research landscape mapped for first time. Available at: https://www.nihr.ac.uk/news/uk-rare-disease-research-landscape-mapped-first-time. Accessed: February 2026.

[3] ABPI. UK must do more on access to rare disease medicines, says ABPI. Available at: https://www.abpi.org.uk/media/news/2025/february/uk-must-do-more-on-access-to-rare-disease-medicines-says-abpi/. Accessed February 2026.

[4] LV / Rare Impact (2024) Sweden – Rare Impact: Challenges and proposals for improving patient access to advanced therapy medicinal products in Sweden. Available at: https://rareimpact.eu/phase-1/challenges-solutions/sweden. Accessed: February 2026.

[5] Cuatrecasas. Health Technology Assessment Royal Decree. Available at: https://www.cuatrecasas.com/en/spain/life-sciences-healthcare/art/health-technology-assessment-royal-decree. Accessed: February 2026.

[6] NICE. Olipudase alfa for treating acid sphingomyelinase deficiency (Niemann–Pick disease) type AB and type B. Available at: https://www.nice.org.uk/guidance/hst32/chapter/1-Recommendations. Accessed: February 2026.

[7] NICE. Vosoritide for treating achondroplasia in people 4 months and over [ID6488]. Available at: https://www.nice.org.uk/guidance/indevelopment/gid-ta11528. Accessed: February 2026.